Gene therapy liver biopsy study
Research type
Research Study
Full title
Liver Biopsy for Gene Therapy in Haemophilia B
IRAS ID
234999
Contact name
Amit Nathwani
Contact email
Sponsor organisation
University College London (UCL)
Eudract number
2018-001333-40
Duration of Study in the UK
1 years, 6 months, 2 days
Research summary
Participants in the AGT4HB study received a single infusion of a modified virus containing a gene for factor IX, a protein that allows the blood to form clots that is present in very low levels in severe haemophilia B, allowing them to produce the missing protein in their livers. Participants in the trial have continued to produce factor IX for an extended period of time, raising the question of whether the virus is present in the liver cells or whether the gene has in fact been integrated into the patient's DNA. This question is of crucial importance in understanding the risks and benefits of gene therapy in haemophilia and we are aiming to collect biopsy samples from the participants of the original AGT4HB and also the GO-8 study. Participants in the GO-8 study receive a single infusion of a modified virus containing a gene for factor VIII, another protein that allows the blood to form clots that is present in very low levels in severe haemophilia A, allowing them to produce the missing protein in their livers.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
18/LO/1487
Date of REC Opinion
29 Oct 2018
REC opinion
Unfavourable Opinion